On February 17, 2022 Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, reported financial results for the fourth quarter and full year 2021 (Press release, Prothena, FEB 17, 2022, View Source [SID1234608258]). In addition, the Company provided 2022 financial guidance and an update on business highlights.
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"Prothena made meaningful progress in multiple therapeutic indications across our portfolio in 2021 with the advancement of three clinical stage programs. We announced the initiation of the confirmatory Phase 3 AFFIRM-AL study of birtamimab, Phase 2b PADOVA study of prasinezumab, and Phase 1 study of PRX005. Additionally, we presented positive preclinical findings for our anti-Aβ PRX012 and our dual Aβ/tau vaccine at AAIC in June last year," said Gene Kinney, Ph.D., President and Chief Executive Officer of Prothena. "In 2021, we also received $200 million from strategic partnerships with leading pharmaceutical companies and bolstered our cash position with $175 million raised through equity offerings. In 2022, we look forward to multiple scientific congresses starting with the presentation of additional preclinical data at AD/PD in March. Our strong capital position funds Prothena through multiple value-creating milestones as we transition into a fully integrated commercial company."
2021 Business Highlights and Upcoming Milestones
Neurodegenerative Diseases Portfolio
Alzheimer’s Disease (AD)
PRX012, a potential best-in-class treatment for AD, is an investigational monoclonal antibody targeting a key epitope at the N-terminus of amyloid beta (Aβ) with high binding potency
•Presented preclinical results at the Alzheimer’s Association International Conference in 2021 (AAIC) demonstrating that PRX012 significantly cleared both pyroglutamate-modified and -unmodified Aβ plaque in post-mortem brain tissue of late-stage AD patients
•Investigational New Drug (IND) application filing expected 1Q 2022
PRX005, a potential best-in-class treatment for AD, is an investigational antibody that specifically targets a key epitope within the microtubule binding region (MTBR) of tau, a protein implicated in diseases including AD, frontotemporal dementia (FTD), progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), and other tauopathies. PRX005 is part of the global neuroscience research and development collaboration with Bristol Myers Squibb
•Received $80 million option payment from Bristol Myers Squibb for execution of U.S. license agreement in 2021
•Phase 1 study initiated in 2021
•Topline Phase 1 data expected in 2022
Dual Aβ/tau vaccine, a potential first-in-class treatment and prevention for AD, is a dual-target vaccine targeting key epitopes within the Aβ and tau proteins to promote amyloid clearance and blockade of pathogenic tau
•AAIC presentation in 2021 showcased preclinical data demonstrating that Prothena’s dual Aβ/tau vaccine generated appropriate and balanced antibody titers promoting both phagocytosis of Aβ plaque and blockade of tau transmission in vitro
•Presentation of preclinical data at the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) expected in March 2022
•IND filing expected in 2023
Parkinson’s Disease (PD)
Prasinezumab, a potential first-in-class treatment for PD, is a humanized monoclonal antibody designed to target key epitopes within the C-terminus of alpha-synuclein and is the focus of the worldwide collaboration with Roche
•Earned $60 million clinical milestone payment in 2021 upon dosing of the first patient in the global Phase 2b PADOVA study for prasinezumab (NCT#04777331)
•Presentation of additional data by Roche at AD/PD expected in March 2022
•Phase 2b PADOVA study results expected in 2024
Rare Peripheral Amyloid Diseases Portfolio
AL Amyloidosis (AL)
Birtamimab, a potential best-in-class amyloid depleter treatment for AL, is an investigational humanized monoclonal antibody designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid that causes organ dysfunction and failure
•Reached Special Protocol Assessment (SPA) agreement with FDA at p≤0.10 and initiated confirmatory Phase 3 AFFIRM-AL study of birtamimab in Mayo Stage IV patients with AL amyloidosis in 2021 (NCT#04973137)
•Confirmatory Phase 3 AFFIRM-AL study results expected in 2024
ATTR Amyloidosis (ATTR)
PRX004, a potential first-in-class treatment for ATTR, is a humanized monoclonal antibody designed to deplete the pathogenic, non-native forms of the TTR protein, and is being developed by Novo Nordisk for the treatment of ATTR cardiomyopathy
•Announced Novo Nordisk acquisition of ATTR business for a total aggregate of up to $1.23 billion and Prothena received $60 million up front payment in 2021
•Novo Nordisk expected to initiate a Phase 2 trial in 1H 2022 with PRX004 for the treatment of ATTR cardiomyopathy
2021 Organizational Highlights
•Sanjiv Patel, MBBS, MA, MBA, appointed to the Board of Directors
•Hideki Garren, M.D., Ph.D., appointed to Chief Medical Officer
•Tran Nguyen, Chief Financial Officer, appointed to the additional, newly created role of Chief Strategy Officer
•Brandon Smith promoted from Chief Business Officer to Chief Operating Officer
Upcoming Investor Conference
Members of the senior management team will present and participate in investor meetings at the following upcoming investor conference:
•Oppenheimer 32nd Annual Healthcare Conference, March 15, 2022, at 1:20 PM ET
Fourth Quarter and Full Year of 2021 Financial Results
For the fourth quarter and full year of 2021, Prothena reported a net loss of $33.2 million and net income of $67.0 million, respectively, as compared to a net loss of $30.7 million and $111.1 million for the fourth quarter and full year of 2020, respectively. Net loss per share for the fourth quarter of 2021 was $0.71 and net income per share on a diluted basis for the full year of 2021 was $1.38, as compared to net loss per share of $0.77 and $2.78 for the fourth quarter and full year of 2020, respectively.
Prothena reported total revenue of $1.2 million and $200.6 million for the fourth quarter and full year of 2021, respectively. Revenue for the fourth quarter of 2021 related to $1.2 million from Bristol Myers Squibb. Revenue for the full year of 2021, included $79.7 million from Bristol Myers Squibb for PRX005 U.S. License and U.S. Development Services and $60.7 million from the sale of the intellectual property and related rights to the Company’s ATTR amyloidosis business and pipeline to Novo Nordisk. In addition, the full year revenue included $60.0 million in clinical milestone payment from Roche related to the global Phase 2b PADOVA study for prasinezumab and a nominal amount of license revenue from Roche. This compares to total revenue of $0.4 million and $0.9 million for the fourth quarter and full year of 2020, primarily from collaboration revenue from Roche.
Research and development (R&D) expenses totaled $22.1 million and $82.3 million for the fourth quarter and full year of 2021, respectively, as compared to $20.8 million and $74.9 million for the fourth quarter and full year of 2020, respectively. The increase in R&D expense for the fourth quarter and full year of 2021 compared to the same periods in the prior year was primarily due to higher personnel expenses, higher clinical trial expenses primarily related to the birtamimab and PRX005 programs (offset in part by lower PRX004 clinical trial expense); offset in part by lower collaboration expenses related to the prasinezumab program with Roche as a result of the cost share opt-out exercised in May 2021 and lower manufacturing costs primarily related to PRX005 and birtamimab programs (offset in part by higher PRX012 preclinical expense). R&D expenses included non-cash share-based compensation expense of $2.9 million and $9.5 million for the fourth quarter and full year of 2021, respectively, as compared to $2.1 million and $8.2 million for the fourth quarter and full year of 2020, respectively.
General and administrative (G&A) expenses totaled $12.2 million and $46.3 million for the fourth quarter and full year of 2021, respectively, as compared to $9.9 million and $38.7 million for the fourth quarter and full year of 2020, respectively. The increase in G&A expenses for the fourth quarter and full year of 2021 compared to the same periods in the prior year was primarily related to higher personnel expenses, legal expenses, consulting and expense for our director and officer insurance premium. G&A expenses included non-cash share-based compensation expense of $4.0 million and $15.1 million for the fourth quarter and full year of 2021, respectively, as compared to $3.2 million and $13.8 million for the fourth quarter and full year of 2020, respectively.
Total non-cash share-based compensation expense was $6.9 million and $24.7 million for the fourth quarter and full year of 2021, respectively, as compared to $5.2 million and $22.0 million for the fourth quarter and full year of 2020.
As of December 31, 2021, Prothena had $580.4 million in cash, cash equivalents and restricted cash, and no debt. This includes net proceeds raised of $175 million raised through equity offerings and a total of $200 million in payments from partners Bristol Myers Squibb, Novo Nordisk and Roche.
As of February 11, 2022, Prothena had approximately 46.7 million ordinary shares outstanding.
2022 Financial Guidance
The Company expects the full year 2022 net cash used in operating and investing activities to be $120 to $132 million, which includes an expected $40 million clinical milestone payment from Novo Nordisk and expects to end the year with approximately $454 million in cash, cash equivalents and restricted cash (midpoint). The estimated full year 2022 net cash used in operating and investing activities is primarily driven by an estimated net loss of $154 to $170 million, which includes an estimated $32 million of non-cash share-based compensation expense.
Conference Call Details
Prothena management will discuss these results and its 2022 financial guidance during a live audio conference call today, Thursday, February 17, 2022, at 4:30 PM ET. The conference call will be made available on the Company’s website at www.prothena.com under the Investors tab in the Events and Presentations section. Following the live audio webcast, a replay will be available on the Company’s website for at least 90 days.
To access the call via dial-in, please dial (888) 440-6385 (U.S. and Canada toll free) or +00 1 646 960-0180 (international) five minutes prior to the start time and refer to conference ID number 92750. A
replay of the call will be available until March 3, 2022, via dial-in at (800) 770-2030 (U.S. toll free) or +00 1 647 362-9199 (international), Conference ID Number 92750.